Gene Therapy for β-Hemoglobinopathies

Molecular Therapy

Volumn 25, Issue 5, 2017, Pages 1142-1154

  Cavazzana, Marina ^a,b,c,d   Antoniani, Chiara ^c,d   Miccio, Annarita ^c,d  

^a HÔPITAL NECKER ENFANTS MALADES   (France)

^b ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

^c INSERM   (France)

^d University Sorbonne Paris Cite   (France)

Author keywords

gene therapy; hematopoietic stem cell; hemoglobinopathies; sickle cell disease; thalassemias

Indexed keywords

BUSULFAN; GRANULOCYTE COLONY STIMULATING FACTOR; HEMOGLOBIN BETA CHAIN; HLA ANTIGEN; LENTIVIRUS VECTOR; PLERIXAFOR;

BCL11A GENE; BETA GLOBIN GENE; BETA THALASSEMIA; CHROMATIN STRUCTURE; DOWN REGULATION; GENE; GENE CONTROL; GENE EDITING; GENE EXPRESSION; GENE LOCUS; GENE MUTATION; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HMGA2 GENE; HUMAN; MOLECULARLY TARGETED THERAPY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REVIEW; SICKLE CELL ANEMIA; ALLOTRANSPLANTATION; CHEMISTRY; CYTOLOGY; DONOR; GAMMARETROVIRUS; GENE VECTOR; GENETICS; HEMATOPOIETIC STEM CELL; IMMUNOLOGY; LENTIVIRUS; METABOLISM; MUTATION; PATHOLOGY; PROCEDURES;

ANEMIA, SICKLE CELL; BETA-GLOBINS; BETA-THALASSEMIA; GAMMARETROVIRUS; GENE EDITING; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HLA ANTIGENS; HUMANS; LENTIVIRUS; MUTATION; TISSUE DONORS; TRANSPLANTATION, HOMOLOGOUS;

EID: 85016445049     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.03.024     Document Type: Review

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